Combination Regimen With Sodium Valproate for Severe Hemophilia: a Single-arm, Phase 1, Pilot Trial.
Study Details
- Hemophilia
- Drug: Sodium valproate extended-release tablets
- Inclusion Criteria:
- Patients with clinically confirmed severe haemophilia;
- Expected survival of ≥ 24 weeks with an ECOG score of 0-2;
- Not having participated in another clinical trial within four weeks;
- Informed consent signed by the patient or an immediate family member.
- Exclusion Criteria:
- Those with other types of blood disorders diagnosed at the morphological or molecular level of the bone marrow;
- Significantly abnormal cardiopulmonary function;
- Hepatic or renal insufficiency;
- Pregnancy or lactation, or inability to use contraception during the trial and for three months before the test and one year after administration
- Persons who are allergic to the drugs likely to be used or where there is a contraindication to their use;
- Those with severe uncontrollable infectious diseases or uncontrolled hypertension, malignancy, etc.;
- Inability to cooperate with a regular follow-up due to psychological, social, family and other geographical circumstances;
- Any other condition that, in the investigator's opinion, makes participation in this trial inappropriate.
Protocol Summary
This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.